Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

NCTID	NCT05092685 (View at clinicaltrials.gov)
Description	Ornithine transcarbamylase deficiency (OTCD) is an inherited metabolic liver disease which means that the body cannot maintain normal levels of ammonia. Ammonia levels can rise (called hyperammonaemic decompensations) which can be life-threatening and may result in impaired neurological development in children. OTCD is a rare genetic disorder characterised by complete or partial lack of the enzyme ornithine transcarbamylase (OTC). (Show More)
Indication	Ornithine Transcarbamylase Deficiency
Compound Name	BGT-OTCD (AAVLK03hOTC)
Sponsor	University College, London
Funder Type	Other
Status	Recruiting
Enrollment Count	12

Target Gene/Variant	OTC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	LK03
Editor Type
Dose 1	6 x 10^11 vg/kg
Dose 2	2 x 10^12 vg/kg
Dose 3	6 x 10^12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United Kingdom

Has US IND	False
Recent Updates	Granted ODD by FDA and EMA